Understanding the challenges, unmet needs, and expectations of mucopolysaccharidoses I, II and VI patients and their caregivers in France: a survey study

BACKGROUND: Mucopolysaccharidoses (MPS) are a group of inherited lysosomal storage diseases caused by defective enzyme activity involved in the catalysis of glycosaminoglycans. Published data on adult patients with MPS remains scarce. Therefore, the present qualitative survey study was aimed at unde...

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Detalles Bibliográficos
Autores principales: Guffon, Nathalie, Genevaz, Delphine, Lacombe, Didier, Le Peillet Feuillet, Eliane, Bausson, Pascale, Noel, Esther, Maillot, François, Belmatoug, Nadia, Jaussaud, Roland
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2022
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9786416/
https://www.ncbi.nlm.nih.gov/pubmed/36564803
http://dx.doi.org/10.1186/s13023-022-02593-2

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